(Boursier.com) — Lysogen has released key results from its Phase 2/3 AAVance study evaluating LYS-SAF302, an investigational gene therapy for the treatment of MPS IIIA, a rare autosomal recessive neurodegenerative lysosomal storage disease that affects approximately 1 in 100,000 newborns.
The study did not reach its primary efficacy endpoint in the main cohort (12 patients recruited at age greater than 30 months) aimed at demonstrating a statistically significant improvement in cognitive DQ 24 months post-treatment, as assessed by BSID-III, compared to the NH cohort. In this cohort, the main secondary efficacy endpoints were not achieved, including the percentage of patients with stabilization or improvement in cognitive AD, AD associated with language and motor functions at 24 months compared to baseline, as well as the evolution of VABS-II scores compared to NH data.
In subjects recruited at an age of less than 30 months, a statistically significant improvement in cognitive development was observed 24 months post-treatment compared to natural history. Subjects in this cohort also achieved key secondary efficacy endpoints. Statistical analysis confirms previously reported qualitative observations in the younger patient population with consistent effects on endpoints.
“Although AAVance did not meet its primary endpoint, the results obtained in the subset of MPS IIIA patients recruited before the age of 30 months are extremely encouraging, as they demonstrate the beneficial effects of the treatment on cognitive development and key secondary endpoints of this devastating childhood disease for which there are no approved disease-modifying treatments,” said Dr. Chester B. Whitley, Professor in the Department of Pediatrics and Experimental Pharmacology. and Clinic at the University of Minnesota, and Principal Investigator of the Lysosomal Disease Network, Minneapolis, MN, USA. “I look forward to continuing the collaboration with Lysogene to analyze the Phase 2/3 data and determine the most appropriate route to bring LYS-SAF302 to the patients most likely to benefit.”
Lysogene considers that these results help define the patient population that could benefit from treatment with LYS-SAF302. The company entered into fruitful discussions with regulatory authorities in the European Union at the end of Q3 2022 and plans to extend these discussions to all relevant regulatory agencies to define a path to rapidly advance the clinical development of LYS. -SAF302 in the population of younger patients with MPS IIIA. The company will finalize the full analysis and evaluation of AAVance’s core cohort data and collaborate with investigators on upcoming results presentations and publications.
In order to pursue the development of LYS-SAF302 in accordance with the requirements of regulatory agencies, Lysogene intends to ensure adequate cash flow. In this regard, strategic discussions are underway, with the priority of a non-dilutive solution in the form of a licensing collaboration for one or more of its programs. In the meantime, the company is maintaining its cost control initiatives launched at the start of the year, which now ensure a cash horizon until February 2023.
©2022 Boursier.com